Phase II study of ipilimumab in adolescents with unresectable stage III or IV malignant melanoma Journal Article
| Authors: | Geoerger, B.; Bergeron, C.; Gore, L.; Sender, L.; Dunkel, I. J.; Herzog, C.; Brochez, L.; Cruz, O.; Nysom, K.; Berghorn, E.; Simsek, B.; Shen, J.; Pappo, A. |
| Article Title: | Phase II study of ipilimumab in adolescents with unresectable stage III or IV malignant melanoma |
| Abstract: | Background Ipilimumab is approved for the treatment of advanced melanoma in adults; however, little information on the efficacy and safety of ipilimumab in younger patients is available. Methods Patients aged 12 to <18 years with previously treated or untreated, unresectable stage III or IV malignant melanoma received ipilimumab 3 or 10 mg/kg every 3 weeks. Primary end-points were 1-year overall survival and safety. Results Over a period of 3.5 years, 12 patients received ipilimumab at either 3 mg/kg (n = 4) or 10 mg/kg (n = 8). The median number of ipilimumab doses was four for 3 mg/kg and three for 10 mg/kg. At 1 year, three of four patients on 3 mg/kg and five of eight patients on 10 mg/kg were alive. Two patients on 10 mg/kg had partial response, and one on 3 mg/kg had stable disease. One patient had durable partial response at 3 years without further treatment, at time of this report. There was one grade 3/4 immune-mediated adverse reaction with 3 mg/kg and five with 10 mg/kg. There were no treatment-related deaths. The study was stopped due to slow accrual. Conclusions At >1 year follow-up, ipilimumab demonstrated activity in melanoma patients aged 12 to <18 years, with a similar safety profile as that seen in adults. Our trial highlights the difficulties of enrolling younger patients with rare diseases in clinical trials for treatments that are approved in adults, suggesting adolescents with cancer types occurring predominantly in adults should be considered for inclusion in adult trials of promising new drugs. Clinical trial registration: NCT01696045. © 2017 Elsevier Ltd |
| Keywords: | adolescent; cancer survival; child; controlled study; major clinical study; overall survival; hepatitis; advanced cancer; dose response; drug dose comparison; drug safety; drug withdrawal; side effect; cancer patient; follow up; ipilimumab; cancer immunotherapy; melanoma; progression free survival; controlled clinical trial; phase 2 clinical trial; nausea; vomiting; cohort analysis; abdominal pain; alanine aminotransferase blood level; aspartate aminotransferase blood level; chill; dyspnea; fever; hyperglycemia; alanine aminotransferase; aspartate aminotransferase; hypokalemia; multicenter study; pancreatitis; pleura effusion; open study; hot flush; inoperable cancer; drug blood level; liver enzyme; cancer control; amylase blood level; drug treatment failure; triacylglycerol lipase blood level; epistaxis; immunopathology; amylase; cholestasis; triacylglycerol lipase; lung lesion; progression-free survival; adolescents; advanced melanoma; acute cholecystitis; infusion related reaction; human; male; female; priority journal; article; adolescent disease; immune-mediated adverse reaction |
| Journal Title: | European Journal of Cancer |
| Volume: | 86 |
| ISSN: | 0959-8049 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2017-11-01 |
| Start Page: | 358 |
| End Page: | 363 |
| Language: | English |
| DOI: | 10.1016/j.ejca.2017.09.032 |
| PROVIDER: | scopus |
| PUBMED: | 29100190 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 4 December 2017 -- Source: Scopus |
